A different approach to Gorlin syndrome

QTORIN^TM rapamycin 3.9% for Gorlin Syndrome

QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin syndrome.

A lifelong risk of malignant skin cancer

Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a serious rare life-long genetic disease caused by a mutation in the PTCH1 gene leading to abherrant hedgehog signaling. Recurrent basal cell carcinomas (BCCs) are a hallmark of this disease.  Individuals with Gorlin syndrome can develop hundreds or even thousands of BCCs, oftentimes beginning in childhood. BCCs are a malignant skin cancer requiring repeated and potentially disfiguring surgical removal for patients with Gorlin syndrome.  Gorlin syndrome affects an estimated 11,000 people in the United States.

There are no FDA approved therapies for Gorlin syndrome.

Targeting Gorlin syndrome

Palvella initiated internal research efforts on the potential for QTORIN™ rapamycin 3.9% in Gorlin syndrome in 2017. QTORIN™ rapamycin 3.9% is designed to inhibit the mTOR pathway, a major driver of BCC tumorigenesis^¹,2. This approach aims to avoid some of the limitations of the direct hedgehog inhibitors, such as resistance and recurrence.

^{Hua, H., Kong, Q., Zhang, H. et al. Targeting mTOR for cancer therapy. J Hematol Oncol 12, 71 (2019) doi:10.1186/s13045-019-0754-1}

^{Kim, A., Back, J. et al. SOX9 Transcriptionally Regulates mTOR-Induced Proliferation of Basal Cell Carcinomas. J Invest Dermatol. 2018 August; 138(8): 1716–1725.}